Israeli Gaucher Association Meeting 2000

The Israeli Gaucher Association Meeting was held on 5
September 2000 in Jerusalem to which delegates from the European Gaucher
Alliance meeting were invited. Dr Gregory Grabowski spoke on 'Summarising 10
years of Enzyme Replacement Therapy' and Prof Ari Zimran on 'New Therapeutic
Approaches to Gaucher Disease'.

Mr Sandy Smith of Genzyme Therapeutics thanked the Israeli
Gauchers Association for the opportunity to address them and said that the year
2000 was the 10th anniversary of enzyme replacement therapy for Gauchers
disease.

The safety and efficacy of the two drugs Ceredase and Cerezyme were
unprecedented. Nearly 3,000 patients are receiving enzyme replacement therapy
in 56 countries. Thanks to the scientists and physicians, patients are leading
full and productive lives and play a valuable role in society.

But more work needs to be done which includes sustained release enzyme,
small molecule therapy (eg substrate deprivation), and gene therapy. He
advocates improved screening of children and early diagnosis.

Dr Chris Moyses said that Oxford GlycoSciences was the first
biotech company founded by Oxford University. The company has 200 employees and
has two strengths: proteomics (discovering which proteins which are over or
under expressed in people cause disease) and drug development.

OGT918 (Vevesca) is on clinical trial for two conditions: Gaucher
Disease and Fabry disease.

Dr Gregory Grabowski, Director of Human Genetics at the
Children's Hospital, Cincinatti, described the status of enzyme replacement
therapy in patients in the last 10 years and spoke about the collaboration of
academics and industry. Progress was also due to the patients participation.

He spoke also about his forthcoming Fosamax trial: Fosamax or placebo,
both with vitamin D 400u and 1500 mg calcium. 82 patients will take part in a
24 month, double blind, trial randomised for age, severity and dose. The
results will be available in 24 months although an interim report should be
available earlier.

Prof Ari Zimran said that even in Israel, many GPs don't see a
single patient with Gauchers disease. Some patients don't hear about treatment
and he gave an example of someone who has severe disease but who felt he did
not meet the criteria for treatment. He also spoke about his research into OGT
918.

Prof Zimran expressed concern about the use of portacaths and the risks
involved. A portacath is a device which is inserted under the skin with a tube
which enters a vein. This may allow easier access for intravenous infusions
than using a peripheral vein n the hand or arm. He said that it is necessary to
have a skilful surgeon to insert one. The device could get infected and it was
important to teach septic care. There was a possibility of getting a thrombosis
(blood clot) and if the portacath gets blocked, it cannot be used and the
patient may need a new one. If the clot gets detached, it could go to the lung.
It is important to flush the portacath regularly with heparin (a drug which
prevents blood clotting).